New hope has been given to people living with Huntingdon’s Disease, a degenerative brain disease caused by a defect in a gene that alters a normal protein in the brain into one that turns on itself and gradually kills the cells. There are around 10,000 people affected by this disease in the UK and until now, has been incurable but this breakthrough in gene therapy may be the beginning of a new kind of research into finding a possible cure for this and other linked brain diseases such as Parkinson’s and Alzheimer’s.
The new drug (Ionis-HTTRx) works by ‘intercepting a messenger molecule and destroying it before the harmful protein can be made, effectively silencing the effects of the mutant gene. It effectively stops Huntingdon’s in its tracks.’
The drug is injected into the spinal fluid each month and so, far has showed no adverse effects on the small number of people taking part in the trial, but importantly it proved to reduce the levels of the disease in the brain.
Roche, the Swiss pharmaceutical company owns the drug and is likely to commence a much larger trial to further test the drug’s effectiveness. Professor of Clinical Neuroscience and Honorary Consultant Neurologist from Cambridge University stated:
“results are encouraging as it shows delivering therapeutic agents of this type is feasible and well tolerated” “the question is whether this is enough to make a difference to patients and their clinical course and for that we will have to wait for bigger trials.”
Despite the encouraging results, some experts are being more cautious at this early stage. University of Edinburgh’s Professor Tar Spires-Jones, UK Dementia Research Institute urged:
“The details of the study have not been peer-reviewed or published yet, so the scientific community simply does not know yet how robust the findings are…With that caveat in mind, the approach used in this trial has excellent potential to prevent Huntingdon’s disease, and if the next stage of larger trials live up to their promise, this will be a breakthrough.”
Extracts taken from: Sarah Knapton, Science Editor, The Telegraph (online) 11 December 2017. Click here for full article.
For more information on Huntingdon's disease click here.